When I think about how many clinical trials it will take before a stem cell therapy is available to patients, I’m reminded of the decades old Tootsie Pop commercial where a kid asks a series of talking animals, “How many licks does it take to get to the Tootsie Roll center of a Tootsie Pop?”
While Mr. Cow, Mr. Fox, and Mr. Turtle are all stumped, Mr. Owl tackles the question like a true scientist:
“A good question. Let’s find out. [Takes Tootsie pop and starts licking]. A One…A Two-hoo…A Three-hee. [Insert loud crunching sounds] A Three!”
The commercial ends with the narrator concluding that the world may never know how many licks it takes to get to the center (because Mr. Owl failed to complete his experiment…not a true scientist after all).
What do Tootsie Pops have to do with stem cell therapies?
I’m not saying that the Tootsie Pop question holds the same level of importance as the question of when scientists will develop a stem cell therapy that cures a disease, but I find it representative of the confusion and uncertainty that the general public has about when the “promise of stem cell research” will become a reality.
Let me explain…
Mr. Owl claims that it only takes three licks to get to the center of a Tootsie Pop, but three licks obviously aren’t enough to get through the hard candy exterior to the chewy tootsie center. According to the Tootsie “Scientific Endeavors” page, “at least three detailed scientific studies” determined that it takes between 144-411 licks to get to the center. My intuition is to go with the scientists, but depending on how the experiment was conducted or maybe the size of the tongue used, the final answer could vary.
Embryonic stem cells
For stem cell clinical trials, the situation is similar. The first clinical trial approved in the U.S. using human embryonic stem cells was in 2009. Since then, hundreds of clinical trials have been conducted globally using pluripotent – either embryonic or induced pluripotent stem cells (iPSCs) – or adult stem cells. But so far, none have made their way routinely to patients outside of a clinical trial setting in the U.S., (although a few stem cell-based products have been approved in other countries), and it’s unclear how many more trials it will take to get to this point.
Part of this murkiness is because we’re still in the early days of stem cell research: human embryonic stem cells were first isolated by James Thomson in 1998, and iPSCs weren’t discovered by Shinya Yamanaka until 2006. Scientists need more time to conduct preclinical research to understand how these stem cells can be best used to treat certain diseases and what stem cells will do when transplanted into patients.
Another other issue is that the U.S. Food and Drug Administration (FDA) has only approved one stem cell therapy – cord blood stem cell transplantation – for commercial use in 2011 and none since then. A big debate is currently ongoing about whether the regulatory landscape needs to change so that stem cell treatments that show promise in trials can get to patients who desperately need them.
Hopefully soon, the FDA will adopt a more efficient strategy for approving stem cell therapies that still keeps patient safety at the forefront. Otherwise it could take a lot longer for newer stem cell technologies like iPSCs to make their way to the clinic (although we’ve seen some encouraging preliminary results using iPSC-based therapy in clinical trials for blindness).
Trial, trial, trial again
So how many clinical trials will it take for a stem cell therapy to succeed sufficiently to gain approval and when will that happen?
Unfortunately, we don’t know the answers to these questions, but we do know that scientists need to continue to develop and test new stem cell treatments in human trials if we want to see any progress.
At CIRM, we are currently funding 16 clinical trials involving stem cell therapies for cancer, heart failure, diabetes, spinal cord injury and other diseases. But we need to fund more trials to increase the odds that some will make it through the gauntlet and prove both safe and effective at treating patients. Our goal now is to fund 50 clinical trials in the next five years. It’s an aggressive plan, but one we feel will hopefully take stem cell therapies from promise to reality.
We also know that CIRM is a soldier in a large army of funding agencies, universities, companies, and scientists around the world battling against time to develop stem cell therapies that could help patients in their lifetimes. And with this stem cell army, we believe we’re getting closer to the chewy center of the Tootsie pop, or in this case, an approved stem cell therapy for patients desperate for a cure.
This blog was written as part of the CCRM Signals iPSC anniversary blog carnival. Please click here to read what other bloggers have to say about the future of stem cells and regenerative medicine.